Implementation of 21st Century Cures Act Expanded Access Policies Requirements

Area of Science:

• Oncology
• Health Policy
• Drug Development

Background:

• The US Food and Drug Administration's (FDA) expanded access pathway enables patients with serious conditions to receive investigational drugs outside clinical trials.
• The 21st Century Cures Act mandates that certain biopharmaceutical companies publicly disclose their expanded access policies.

Purpose of the Study:

• To assess the implementation of the 21st Century Cures Act requirements for public expanded access policies among US biopharmaceutical companies with oncology drugs.
• To determine the completeness of information within disclosed expanded access policies.

Main Methods:

• A cross-sectional study characterizing US biopharmaceutical companies with oncology-related investigational drugs.
• Analysis of publicly available expanded access policies for compliance with the 21st Century Cures Act disclosure requirements.

Main Results:

• Only 32% (140/423) of applicable companies have a public expanded access policy.
• Less than one-third (31%) of these policies contain all information mandated by the Act.
• Larger companies and those with FDA expedited designations or approved drugs were significantly more likely to have public policies.

Conclusions:

• The 21st Century Cures Act's impact on timely medical decisions and information access for expanded access to investigational oncology therapies appears limited.
• Smaller and newer companies are less likely to provide public expanded access policies, potentially widening informational gaps for patients and physicians.